79. 家族性高コレステロール血症（ホモ接合体） ［臨床試験数：54，薬物数：69（DrugBank：24），標的遺伝子数：6，標的パスウェイ数：14］
Searched query = "Homozygous familial hypercholesterolemia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04034485 | November 7, 2019 | 11 November 2019 | Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH | Randomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy | Homozygous Familial Hypercholesterolemia | Drug: LIB003;Drug: evolocumab | LIB Therapeutics LLC | Recruiting | 10 Years | N/A | All | 70 | Phase 3 | United States | |
| 2 | NCT04080050 | September 30, 2019 | 11 November 2019 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy | Regenxbio Inc. | Recruiting | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United States;Canada;Italy;Netherlands | |
| 3 | NCT04031742 | August 12, 2019 | 29 July 2019 | A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia | Phase 2b/3 Study to Assess the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia. Part 1 - Open-label, Two-arm, Multicenter Pilot Study to Evaluate Efficacy and Safety of IBI 306 in Subjects With Homozygous Familial Hypercholesterolemia. Part 2 - Open-label, Single-arm, Multicenter Study to Evaluate Efficacy and Safety of IBI 306 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Biological: IBI306 | Innovent Biologics (Suzhou) Co. Ltd. | Not recruiting | 18 Years | 80 Years | All | 30 | Phase 2/Phase 3 | ||
| 4 | NCT03814187 | April 16, 2019 | 4 November 2019 | Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C | An Open-label Extension Trial of the Phase III Lipid-lowering Trials to Assess the Effect of Long Term Dosing of Inclisiran Given as Subcutaneous Injections in Subjects With High Cardiovascular Risk and Elevated LDL-C | ASCVD;Elevated Cholesterol;Heterozygous Familial Hypercholesterolemia;Homozygous Familial Hypercholesterolemia | Drug: Inclisiran Sodium | The Medicines Company | Recruiting | 18 Years | N/A | All | 3700 | Phase 3 | United States;Canada;Czechia;Denmark;Germany;Hungary;Netherlands;Poland;South Africa;Spain;Sweden;Ukraine;United Kingdom | |
| 5 | NCT03933293 | April 2019 | 13 May 2019 | A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH | A Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: AK102;Drug: Statins;Drug: Ezetimibe | Akeso | Not recruiting | 18 Years | N/A | All | 59 | Phase 2 | China | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03851705 | February 6, 2019 | 4 March 2019 | A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | A Two-Part (Double-Blind Placebo Controlled/Open-Label) Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Subjects With Homozygous Familial Hypercholesterolemia (Hofh) (ORION-5) | Renal Impairment | Drug: Inclisiran for injection;Drug: Placebos | The Medicines Company | Recruiting | 18 Years | 80 Years | All | 45 | Phase 2/Phase 3 | Czechia;Hong Kong;Russian Federation;Serbia;Taiwan;Ukraine | |
| 7 | JPRN-JapicCTI-184099 | 30/9/2018 | 10 September 2019 | STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | Homozygous familial hypercholesterolemia (HoFH) | Intervention name : Evinacumab INN of the intervention : Evinacumab Dosage And administration of the intervention : INTRAVENOUS Injection Control intervention name : PLACEBO INN of the control intervention : - Dosage And administration of the control intervention : INTRAVENOUS Injection | Regeneron Pharmaceuticals, Inc.(ICCC: PAREXEL International) | Not Recruiting | 18 | BOTH | 57 | NA | Japan, North America, Europe, Oceania, Africa | ||
| 8 | EUCTR2017-002297-39-NO | 19/09/2018 | 28 February 2019 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0 Level: PT Classification code 10020603 Term: Hypercholesterolaemia System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 Pharmaceutical Form: Solution for injection in pre-filled syringe INN or Proposed INN: Alirocumab CAS Number: 1245916-14-6 Current Sponsor code: SAR236553 (RGN727) Other descriptive name: ALIROCUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 75- Pharmaceutical form of the placebo: Solution for infusion in pre-filled syringe Route of administration of the placebo: Subcutaneous use Product Name: Alirocumab Product Code: SAR236553 Pharmaceutical Form: Solution for injection in pre-filled syringe INN or Proposed INN: Alirocumab CAS Number: 1245916-14-6 Current Sponsor code: SAR236553 (RGN727) Other descriptive name: ALIROCUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 150- Pharmaceutical form of the placebo: Solution for infusion in pre-filled syringe Route of administration of the placebo: Subcutaneous use | Sanofi-Aventis Recherche & Développement | Authorised | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway | |||
| 9 | NCT03510715 | August 31, 2018 | 14 October 2019 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia | Hypercholesterolemia | Drug: Alirocumab SAR236553 (REGN727);Drug: Rosuvastatin;Drug: Ezetimibe;Drug: Cholestyramine;Drug: Nicotinic acid;Drug: Fenofibrate;Drug: Omega-3 fatty acids;Drug: Atorvastatin;Drug: Simvastatin;Drug: Fluvastatin;Drug: Pravastatin;Drug: Lovastatin | Sanofi | Regeneron Pharmaceuticals | Not recruiting | 8 Years | 17 Years | All | 18 | Phase 3 | Brazil;Canada;Denmark;Mexico;Netherlands;Russian Federation;Slovenia;Spain;Taiwan;Turkey;Austria;France;Italy;Norway;United States |
| 10 | NCT03403374 | August 4, 2018 | 28 October 2019 | Safety and Tolerability of Repatha in Indian Subjects With Homozygous Familial Hypercholesterolemia | A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India | Homozygous Familial Hypercholesterolemia HoFH | Drug: Repatha® (evolocumab) | Amgen | Not recruiting | 12 Years | 80 Years | All | 30 | Phase 4 | India | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03455777 | April 12, 2018 | 10 December 2018 | Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: AKCEA-ANGPTL3-LRX | Akcea Therapeutics | Ionis Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | All | 0 | Phase 2 | Canada |
| 12 | NCT03409744 | March 13, 2018 | 26 August 2019 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: evinacumab | Regeneron Pharmaceuticals | Recruiting | 12 Years | N/A | All | 100 | Phase 3 | United States;Australia;Canada;France;Greece;Italy;Netherlands;Norway;Ukraine | |
| 13 | NCT03156621 | October 3, 2017 | 17 June 2019 | Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: Alirocumab;Drug: Placebo | Regeneron Pharmaceuticals | Sanofi | Recruiting | 18 Years | N/A | All | 74 | Phase 3 | Germany;Greece;Italy;Japan;South Africa;Turkey;Ukraine;Austria;Canada;Czechia;France;United States |
| 14 | EUCTR2017-001388-19-FR | 28 February 2019 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1 Level: LLT Classification code 10020604 Term: Hypercholesterolemia System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: Evinacumab CAS Number: 1446419-85-7 Current Sponsor code: REGN1500 Other descriptive name: EVINACUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Powder and solvent for solution for injection Route of administration of the placebo: Intravenous use Trade Name: Praluent Pharmaceutical Form: Solution for injection in pre-filled pen INN or Proposed INN: ALIROCUMAB CAS Number: 1245916-14-6 Other descriptive name: ALIROCUMAB Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- | Regeneron Pharmaceuticals Inc. | Not Available | Female: yes Male: yes | 57 | Phase 3 | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands | ||||
| 15 | EUCTR2017-003170-13-FR | 28 February 2019 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1 Level: LLT Classification code 10020604 Term: Hypercholesterolemia System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: Evinacumab CAS Number: 1446419-85-7 Current Sponsor code: REGN1500 Other descriptive name: EVINACUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 150- Trade Name: Praluent Pharmaceutical Form: Solution for injection in pre-filled pen INN or Proposed INN: ALIROCUMAB CAS Number: 1245916-14-6 Other descriptive name: ALIROCUMAB Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- | Regeneron Pharmaceuticals, Inc. | Not Available | Female: yes Male: yes | 100 | Phase 3 | United States;Greece;Ukraine;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Netherlands;Germany;Japan | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT02963311 | December 13, 2016 | 11 June 2018 | A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: ALN-PCSSC;Drug: Standard of Care | The Medicines Company | Not recruiting | 12 Years | N/A | All | 4 | Phase 2 | United States;Netherlands;South Africa | |
| 17 | EUCTR2015-004003-23-IT | 28/09/2016 | 12 March 2018 | A controlled study to assess the safety, tolerability and activity of the study drug ISIS 703802 given subcutaneously to Healthy Volunteers with high triglycerides and patients with Familial Hypercholesterolemia | A Placebo-Controlled, Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of ISIS 703802, Targeting ANGPTL3, Administered Subcutaneously to Healthy Volunteers with Elevated Triglycerides and Subjects with Familial Hypercholesterolemia | Heterozygous familial hypercholesterolemia and Homozygous familial hypercholesterolemia MedDRA version: 19.0 Level: LLT Classification code 10054380 Term: Familial hypercholesterolemia System Organ Class: 100000004850 MedDRA version: 19.0 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 100000004850 MedDRA version: 19.0 Level: LLT Classification code 10057079 Term: Heterozygous familial hypercholesterolemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: ISIS 703802 Product Code: ISIS 703802 Pharmaceutical Form: Solution for injection INN or Proposed INN: ISIS 703802 Current Sponsor code: ISIS 703802 Other descriptive name: ISIS 703802 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | Ionis Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 61 | Phase 1;Phase 2 | Canada;Netherlands;Italy | |||
| 18 | NCT02624869 | September 10, 2016 | 22 October 2019 | Open Label Study to Evaluate Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Pediatric Subjects (10 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH). | Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) | Familial Hypercholesterolemia | Biological: evolocumab (AMG 145) | Amgen | Recruiting | 10 Years | 17 Years | All | 115 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Canada;Colombia;Czechia;Greece;Hungary;Italy;Malaysia;Netherlands;Norway;Poland;Portugal;Slovenia;South Africa;Spain;Switzerland;Turkey;United Kingdom | |
| 19 | EUCTR2016-000411-32-NL | 15/07/2016 | 1 October 2018 | A study to the safety and efficacy of REGN1500 in patients with Hereditary abnormal (high) cholesterol level | AN OPEN-LABEL, SINGLE-ARM, PROOF-OF-CONCEPT STUDY TO EVALUATE THE SAFETY AND EFFICACY OF SINGLE AND MULTIPLE DOSES OF REGN1500 IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | Homozygous Familial Hypercholesterolemia MedDRA version: 19.0 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: evinacumab Product Code: REGN1500 Pharmaceutical Form: Lyophilisate for solution for infusion INN or Proposed INN: EVINACUMAB CAS Number: 1446419-85-7 Current Sponsor code: REGN1500 Other descriptive name: EVINACUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Product Name: Evinacumab Product Code: REGN1500 Pharmaceutical Form: Lyophilisate for solution for injection INN or Proposed INN: EVINACUMAB CAS Number: 1446419-85-7 Current Sponsor code: REGN1500 Other descriptive name: EVINACUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Product Name: evinacumab Product Code: REGN1500 Pharmaceutical Form: Lyophilisate for solution for infusion INN or Proposed INN: EVINACUMAB CAS Number: 1446419-85-7 Current Sponsor code: REGN1500 Other descriptive name: EVINACUMAB Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Regeneron Pharmaceuticals, Inc. | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | United States;Canada;Netherlands | |||
| 20 | EUCTR2014-003806-33-GR | 30/06/2016 | 8 August 2016 | A clinical study in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy conducted to assess safety, tolerability and efficacy of Lomitapide | A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | Homozygous familial hypercholesterolemia (HoFH). MedDRA version: 18.1 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lomitapide Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard INN or Proposed INN: LOMITAPIDE CAS Number: 202914-84-9 Current Sponsor code: AEGR-733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2- Product Name: lomitapide Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard INN or Proposed INN: LOMITAPIDE CAS Number: 202914-84-9 Current Sponsor code: AEGR-733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Product Name: lomitapide Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard INN or Proposed INN: LOMITAPIDE CAS Number: 202914-84-9 Current Sponsor code: AEGR 733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- Product Name: lomitapide Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard INN or Proposed INN: LOMITAPIDE CAS Number: 202914-84-9 Current Sponsor code: AEGR-733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- | Aegerion Pharmaceuticals Inc. | Not Recruiting | Female: yes Male: yes | 45 | Phase 3 | Canada;Greece;Turkey;Austria;Israel;Germany;Italy | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | EUCTR2015-002276-25-AT | 27/06/2016 | 28 February 2019 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0 Level: LLT Classification code 10057100 Term: Homozygous familial hypercholesterolaemia System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10057099 Term: Heterozygous familial hypercholesterolaemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 Pharmaceutical Form: Solution for injection in pre-filled pen INN or Proposed INN: EVOLOCUMAB Current Sponsor code: AMG 145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 140- Pharmaceutical form of the placebo: Solution for injection in pre-filled pen Route of administration of the placebo: Subcutaneous use Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 Pharmaceutical Form: Solution for injection in cartridge INN or Proposed INN: EVOLOCUMAB Current Sponsor code: AMG 145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 120- | Amgen Inc | Authorised | Female: yes Male: yes | 115 | Phase 3 | Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;United Kingdom;Hungary;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;South Africa;Netherlands;Germany;New Zealand | |||
| 22 | JPRN-UMIN000022603 | 2016/06/06 | 2 April 2019 | Non-randomized, prospective, non-controlled study of changes of lipid parameters after introduction of evolocumab in patients with heterozygous familial hypercholesterolemia under LDL-apheresis therapy. | Homozygous familial hypercholesterolemia | Subcutaneous injection of evolocumab | Kanazawa University Hospital | Not Recruiting | 20years-old | 100years-old | Male and Female | 2 | Not selected | Japan | ||
| 23 | NCT02722408 | May 2016 | 16 December 2017 | Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1) | A Phase 2 Open-Label, Dose-Finding Study to Assess the Efficacy, Safety, and Tolerability of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid Lowering Therapy (COBALT-1) | Hypercholesteremia | Drug: Gemcabene 300 mg;Drug: Gemcabene 600 mg;Drug: Gemcabene 900 mg | Gemphire Therapeutics, Inc. | Not recruiting | 17 Years | N/A | All | 8 | Phase 2 | United States;Canada;Israel | |
| 24 | NCT02765841 | May 2016 | 5 March 2018 | Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | Not recruiting | 5 Years | 17 Years | All | 0 | Phase 3 | ||
| 25 | NCT02651675 | March 2016 | 30 September 2019 | A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Biological: AAV directed hLDLR gene therapy | Regenxbio Inc. | National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United States;Canada;Italy;Netherlands |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT02434497 | June 6, 2015 | 12 March 2018 | A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia | An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Rosuvastatin 20mg | AstraZeneca | Not recruiting | 6 Years | 18 Years | All | 9 | Phase 3 | Belgium;Canada;Denmark;Israel;Malaysia;Taiwan | |
| 27 | NCT02399852 | June 2015 | 8 January 2018 | Effects of Lomitapide on Carotid and Aortic Atherosclerosis | Effects of Lomitapide on Carotid and Aortic Atherosclerosis in Patients Treated With Lomitapide in Usual Care (CAPTURE) | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | All | 0 | N/A | ||
| 28 | EUCTR2014-004856-68-NO | 29/04/2015 | 3 October 2016 | A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MBX-8025 Product Code: MBX-8025 Pharmaceutical Form: Capsule INN or Proposed INN: MBX-8025 CAS Number: 928821-40-3 Current Sponsor code: MBX-8025 Concentration unit: mg milligram(s) Concentration type: range Concentration number: 50-100 Pharmaceutical form of the placebo: Capsule Route of administration of the placebo: Oral use | CymaBay Therapeutics, Inc. | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | France;Netherlands;Norway | |||
| 29 | NCT02472535 | April 2015 | 11 April 2016 | Study to Evaluate the Effects of MBX-8025 in Patients With HoFH | A 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Other: Run-In Period: Placebo;Drug: MBX-8025 50 mg (Dose Escalation Period 1);Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2);Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3) | CymaBay Therapeutics, Inc. | Not recruiting | 18 Years | N/A | Both | 13 | Phase 2 | Canada;France;Netherlands;Norway | |
| 30 | EUCTR2014-004746-99-BE | 23/02/2015 | 9 January 2017 | A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia. | An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH).;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Film-Coated Tablet Product Name: Crestor Product Code: ZD4522 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Rosuvastatin calcium CAS Number: 147098-20-2 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- | AstraZeneca AB | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT02265952 | February 4, 2015 | 20 August 2018 | Study of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: REGN1500 | Regeneron Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 9 | Phase 2 | United States;Canada;Netherlands | |
| 32 | NCT02226198 | November 2014 | 19 October 2017 | A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia | A Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Rosuvastatin 20mg;Drug: Placebo | AstraZeneca | Not recruiting | 6 Years | 17 Years | All | 20 | Phase 3 | Belgium;Canada;Denmark;Israel;Malaysia;Netherlands;Taiwan;Germany;Lebanon;Sweden;United States | |
| 33 | EUCTR2014-000972-24-SE | 20/08/2014 | 14 September 2015 | A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia | A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1 Level: LLT Classification code 10054380 Term: Familial hypercholesterolemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: calcium rosuvastatin CAS Number: 147098-20-2 Current Sponsor code: ZD4522 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use | AstraZeneca AB | Not Recruiting | Female: yes Male: yes | 25 | United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden | ||||
| 34 | NCT02173158 | April 2014 | 29 June 2015 | Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy | A Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy | Familial Hypercholesterolemia - Homozygous | Drug: lomitapide | Aegerion Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | Both | 10 | Phase 3 | Japan | |
| 35 | NCT02135705 | March 2014 | 8 January 2018 | LOWER: Lomitapide Observational Worldwide Evaluation Registry | LOWER: Lomitapide Observational Worldwide Evaluation Registry | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | Recruiting | 18 Years | N/A | All | 300 | N/A | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2012-002434-37-GB | 23/05/2013 | 28 February 2019 | n/a | A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients with Homozygous Familial Hypercholesterolemia (HoFH) with a 52-Week Open-Label Extension - Anacetrapib in Patients with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MK-0859 Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’- Pharmaceutical Form: Tablet Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc | Not Recruiting | Female: yes Male: yes | 45 | Phase 3 | United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;Italy;United Kingdom | |||
| 37 | EUCTR2011-005400-15-GB | 22/05/2013 | 28 February 2019 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Familial Hypercholesterolemia MedDRA version: 19.0 Level: LLT Classification code 10057100 Term: Homozygous familial hypercholesterolaemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Pharmaceutical Form: Solution for injection Current Sponsor code: AMG 145 Other descriptive name: AMG 145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 70- Product Name: AMG 145 Pharmaceutical Form: Solution for injection in pre-filled pen Current Sponsor code: AMG 145 Other descriptive name: AMG145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 140- Product Name: AMG 145 Pharmaceutical Form: Solution for injection in cartridge Current Sponsor code: AMG 145 Other descriptive name: AMG145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 120- | Amgen Inc | Not Recruiting | Female: yes Male: yes | 300 | Phase 2;Phase 3 | United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan | |||
| 38 | NCT02286596 | April 2013 | 11 April 2016 | Comparisons of Two Low-density Lipoprotein Apheresis Systems in Patients With Homozygous Familial Hypercholesterolemia | Comparisons of Two Low-density Lipoprotein Apheresis Systems in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Device: heparin-induced extracorporeal LDL precipitation;Device: dextran sulfate adsorption | Laval University | Not recruiting | 18 Years | 60 Years | Both | 9 | N/A | Canada | |
| 39 | EUCTR2011-005400-15-BE | 22/03/2012 | 28 May 2018 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Familial Hypercholesterolemia MedDRA version: 20.0 Level: LLT Classification code 10057100 Term: Homozygous familial hypercholesterolaemia System Organ Class: 100000012386 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Pharmaceutical Form: Solution for injection Current Sponsor code: AMG 145 Other descriptive name: AMG 145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 70- Product Name: AMG 145 Pharmaceutical Form: Solution for injection in pre-filled pen Current Sponsor code: AMG 145 Other descriptive name: AMG145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 140- Product Name: AMG 145 Pharmaceutical Form: Solution for injection in cartridge Current Sponsor code: AMG 145 Other descriptive name: AMG145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 120- | Amgen Inc | Not Recruiting | Female: yes Male: yes | 300 | Phase 2;Phase 3 | United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan | |||
| 40 | EUCTR2011-005399-40-BE | 09/03/2012 | 21 August 2017 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolaemia MedDRA version: 14.1 Level: LLT Classification code 10057100 Term: Homozygous familial hypercholesterolaemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Pharmaceutical Form: Solution for injection in pre-filled pen Current Sponsor code: AMG 145 Other descriptive name: AMG 145 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 140- Pharmaceutical form of the placebo: Solution for injection in pre-filled pen Route of administration of the placebo: Subcutaneous use | Amgen Inc | Not Recruiting | Female: yes Male: yes | 59 | Phase 2;Phase 3 | United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;South Africa;Netherlands;New Zealand | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | NCT01412034 | November 2011 | 19 February 2015 | Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects | Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: CER-001 | Cerenis Therapeutics, SA | Not recruiting | 12 Years | N/A | Both | 23 | Phase 2 | United States;Canada;Italy;Netherlands;United Kingdom | |
| 42 | EUCTR2010-023742-79-IT | 25/03/2011 | 12 October 2015 | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND | Homozygous Familial Hypercholesterolemia MedDRA version: 9.1 Level: LLT Classification code 10057080 | Product Name: Lomitapide Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard Current Sponsor code: AEGR-733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Product Name: Lomitapide Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard Current Sponsor code: AEGR-733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- | AEGERION PHARMACEUTICALS | Not Recruiting | Female: yes Male: yes | 23 | Phase 3 | Italy | |||
| 43 | NCT00943306 | October 29, 2009 | 11 June 2018 | Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia | Familial Hypercholesterolemia | Drug: lomitapide | Aegerion Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | All | 19 | Phase 3 | United States;Canada;Italy;South Africa | |
| 44 | EUCTR2008-007058-36-IT | 25/06/2009 | 19 March 2012 | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND | Homozygous Familial Hypercholesterolemia MedDRA version: 9.1 Level: LLT Classification code 10048373 Term: Hypercholesterolaemia aggravated | Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Product Code: AEGR-733 Pharmaceutical Form: Capsule, hard Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- | Aegerion Pharmaceuticals | Not Recruiting | Female: yes Male: yes | 25 | Phase 3 | Italy | |||
| 45 | NCT00730236 | December 2007 | 19 October 2017 | A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH) | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy | Homozygous Familial Hypercholesterolemia | Drug: AEGR-733 | Aegerion Pharmaceuticals, Inc. | FDA Office of Orphan Products Development | Not recruiting | 18 Years | N/A | All | 29 | Phase 3 | United States;Canada;Italy;South Africa |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | EUCTR2005-003449-15-GB | 12/07/2007 | 10 July 2015 | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects - RADICHOL I | Reduction of low density lipoprotein C (LDL-C) in Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 9.1 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia | Product Name: ISIS 301012 Product Code: ISIS 301012 Pharmaceutical Form: Solution for injection Current Sponsor code: ISIS 301012 Other descriptive name: ISIS 301012 phosphorothioate oligonucleotide Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | Genzyme Europe B.V. | Authorised | Female: yes Male: yes | 50 | United Kingdom | ||||
| 47 | EUCTR2005-003626-26-GB | 14/06/2006 | 18 April 2012 | A double-blind, placebo-controlled, randomized study to evaluate the efficacy and safety of TAK-475 or placebo when co-administered with current lipid-lowering therapy in subjects with homozygous familial hypercholesterolemia | A double-blind, placebo-controlled, randomized study to evaluate the efficacy and safety of TAK-475 or placebo when co-administered with current lipid-lowering therapy in subjects with homozygous familial hypercholesterolemia | Treatment of primary dyslipidemia MedDRA version: 8 Level: PT Classification code 10058108 | Product Name: TAK-475 Product Code: TAK-475 Pharmaceutical Form: Tablet Current Sponsor code: TAK-475 Concentration unit: % percent Concentration type: range Concentration number: 95-105 Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Takeda Europe R&D Centre Ltd | Not Recruiting | Female: yes Male: yes | 40 | United Kingdom | ||||
| 48 | NCT00704535 | March 2006 | 19 October 2017 | Evaluation of the Safety, Tolerability and Efficacy of Ezetimibe on a Select Population of Filipinos With Hypercholesterolemia (Study P04748)(COMPLETED) | Post-marketing Surveillance of the Safety, Tolerability and Efficacy of Ezetimibe Among Filipino Patients | Primary Hypercholesterolemia;Homozygous Familial Hypercholesterolemia | Drug: Ezetimibe | Merck Sharp & Dohme Corp. | Not recruiting | 18 Years | N/A | All | 4105 | N/A | Philippines | |
| 49 | NCT00280995 | January 2006 | 15 August 2016 | Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy | A Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | Hypercholesterolemia, Familial | Drug: ISIS 301012 | Kastle Therapeutics, LLC | Ionis Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | Both | 12 | Phase 2 | United States;Netherlands |
| 50 | NCT00079846 | September 2003 | 19 February 2015 | Implitapide in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy | Safety and Efficacy Study of Implitapide Compared With Placebo in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy | Familial Hypercholesterolemia | Drug: Implitapide | Medical Research Laboratories International | Not recruiting | 8 Years | 70 Years | Both | 60 | Phase 2 | United States;Canada;Israel;Netherlands;Norway | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT01556906 | June 2003 | 19 October 2017 | Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor | A Phase II Open Label, Dose-Escalation Study to Determine the Safety, Tolerability and Efficacy of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor BMS-201038 in Patients With Homozygous Familial Hypercholeterolemia | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | University of Pennsylvania;Doris Duke Charitable Foundation | Not recruiting | 13 Years | N/A | All | 6 | Phase 2 | United States |
| 52 | NCT00092833 | July 2002 | 16 December 2017 | Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED) | An Open-Label, Worldwide, Treatment Use Study to Provide Ezetimibe 10 Mg/Day to Patients With Homozygous Familial Hypercholesterolemia or Homozygous Sitosterolemia | Hypercholesterolemia, Familial;Lipid Metabolism, Inborn Errors | Drug: Comparator: ezetimibe | Merck Sharp & Dohme Corp. | Not recruiting | 8 Years | N/A | All | 49 | Phase 3 | United States | |
| 53 | NCT03885921 | October 25, 2000 | 1 April 2019 | Safety and Tolerability Study of Ezetimibe (SCH 058235/MK-0653) Plus Atorvastatin or Simvastatin in Homozygous Familial Hypercholesterolemia (P01417/MK-0653-019) | Long-Term, Open-Label, Safety and Tolerability Study of SCH 58235 in Addition to Atorvastatin or Simvastatin in the Therapy of Homozygous Familial Hypercholesterolemia | Hypercholesterolemia | Drug: Ezetimibe;Drug: Atorvastatin;Drug: Simvastatin | Merck Sharp & Dohme Corp. | Not recruiting | 12 Years | N/A | All | 44 | Phase 3 | ||
| 54 | NCT03884452 | May 3, 2000 | 24 June 2019 | Ezetimibe (SCH 58235) Taken With Either Atorvastatin or Simvastatin in Participants With Familial Hypercholesterolemia (MK-0653-018) | A Phase III Efficacy And Safety Study of Ezetimibe (SCH58235) 10 mg in Addition to Atorvastatin or Simvastatin in the Therapy of Homozygous Familial Hypercholesterolemia | Familial Hypercholesterolemia | Drug: Atorvastatin;Drug: Simvastatin;Drug: Ezetimibe;Drug: Placebo for Ezetimibe | Merck Sharp & Dohme Corp. | Not recruiting | 12 Years | N/A | All | 50 | Phase 3 |